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An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials
Inherited retinal dystrophies (IRDs) are a group of rare eye diseases caused by gene mutations that result in the degradation of cone and rod photoreceptors or the retinal pigment epithelium. Retinal degradation progress is often irreversible, with clinical manifestations including color or night bl...
Autores principales: | Chiu, Wei, Lin, Ting-Yi, Chang, Yun-Chia, Isahwan-Ahmad Mulyadi Lai, Henkie, Lin, Shen-Che, Ma, Chun, Yarmishyn, Aliaksandr A., Lin, Shiuan-Chen, Chang, Kao-Jung, Chou, Yu-Bai, Hsu, Chih-Chien, Lin, Tai-Chi, Chen, Shih-Jen, Chien, Yueh, Yang, Yi-Ping, Hwang, De-Kuang |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8123696/ https://www.ncbi.nlm.nih.gov/pubmed/33926102 http://dx.doi.org/10.3390/ijms22094534 |
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